Bronchitol effective in high-risk bronchiectasis patients

Pharmaxis (ASX:PXS) says new analysis of an unsuccessful phase III trial of Bronchitol in bronchiectasis shows a greater effect in higher risk patients.

The phase III trial, which reported last year, did not meet its primary endpoint of a statistically significant reduction in pulmonary exacerbation rates over a 12-month treatment period.

But new analysis of the results examined a higher risk subgroup of patients who continued to suffer breathlessness even at rest, despite receiving the best standard of care.

Data from the analysis is being presented by Dr Anthony de Soyza, a senior lecturer in respiratory medicine at Newcastle University UK, at the European Respiratory Society Meeting.

The data show that Bronchitol demonstrated clinically significant improvements in exacerbation rate, antibiotic use and scores for respiratory health among this subgroup.

“Bronchiectasis continues to be a disease with very few treatment options and showing a clear treatment effect in such a heterogeneous patient population has proved difficult,” Pharmaxis CEO Gary Philipps commented. “The benefit shown in this subgroup... is therefore quite interesting.”

Pharmaxis decided not to proceed with a regulatory submission for Bronchitol in bronchectasis based on the initial failure to achieve the primary endpoint.

Philipps said that while Pharmaxis “is not currently planning to progress the... data with another study, the authors of this new analysis noted that the positive finding should encourage further investigation”.

Bronchitol is a spray-dried formulation of mannitol approved for the treatment of cystic fibrosis in Australia and Europe.

Pharmaxis (ASX:PXS) shares were trading unchanged at $0.055 as of around 1 pm on Tuesday.